CRISPR-Cas9 in acute myeloid leukaemia: Current state-of-art and future perspectives

IF 5.5 3区医学 Q1 PHARMACOLOGY & PHARMACY

Current Opinion in Pharmacology Pub Date : 2025-12-01 Epub Date: 2025-10-16 DOI:10.1016/j.coph.2025.102582

Federico Zingarelli , Jacopo Nanni , Gianluca Cristiano , Letizia Zannoni , Antonio Curti

引用次数: 0

Abstract

CRISPR-Cas9 gene editing technology has gained attention as a new, reliable and manageable tool for the treatment of previously incurable monogenic diseases. Besides exciting results in this setting, ethical, safety and crucial technical issues have not been fully clarified. More importantly, the role of this potent editing tool in the context of a genetically complex and heterogeneous hematologic malignancy such as acute myeloid leukemia (AML) has not yet been defined to date. In this review we aim to summarize and exploring the ultimate CRISPR-cas9 based strategies for diagnosis, risk stratification and treatment in the context of AML.

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CRISPR-Cas9在急性髓性白血病中的应用：现状和未来展望

CRISPR-Cas9基因编辑技术作为治疗以前无法治愈的单基因疾病的一种新的、可靠的、可管理的工具而受到关注。除了令人兴奋的结果，伦理、安全和关键的技术问题还没有完全澄清。更重要的是，这种强大的编辑工具在遗传复杂和异质性血液恶性肿瘤（如急性髓性白血病（AML））的背景下的作用迄今尚未确定。在这篇综述中，我们旨在总结和探索基于CRISPR-cas9的AML诊断、风险分层和治疗的最终策略。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Opinion in Pharmacology 医学-药学

CiteScore

8.80

自引率

2.50%

发文量

131

审稿时长

4-8 weeks

期刊介绍： Current Opinion in Pharmacology (COPHAR) publishes authoritative, comprehensive, and systematic reviews. COPHAR helps specialists keep up to date with a clear and readable synthesis on current advances in pharmacology and drug discovery. Expert authors annotate the most interesting papers from the expanding volume of information published today, saving valuable time and giving the reader insight on areas of importance.